Tolerability Study of Xerecept® in Pediatric Patients

Phase 1

Terminated

• Conditions: Brain Edema; Brain Tumor
• Interventions: Drug: XERECEPT

• Registration Number: NCT01369121
• Lead Sponsor: Celtic Pharma Development Services

Brief Summary

This study is being conducted to evaluate the safety and tolerability of Xerecept® in children with central nervous system tumors and to identify appropriate doses of Xerecept® to be used in subsequent pediatric clinical trials.

Detailed Description

Steroid-related side effects are much more severe in a pediatric population than they are in adults. The side effects appear more quickly and are often apparent in as few as 14 days. Dexamethasone side effects in children include rounding of the face, acne, increased body hair, muscle weakness, osteoporosis, mood changes, weight gain, fluid retention, glucose instability, high blood pressure, increased susceptibility to infection, stunted growth and aseptic necrosis of the hip joints14-16.

Although extensive studies have been performed with Xerecept® in adults, no studies have been performed with Xerecept® in pediatric subjects. Therefore, this study is being conducted to evaluate the safety and tolerability of Xerecept® in children with central nervous system tumors and to identify appropriate doses of Xerecept® to be used in subsequent pediatric clinical trials. The study will also explore if Xerecept®, when administered to pediatric patients, is effective in allowing decreases in dexamethasone dosing

Study Timeline

• Study Start: 2010-06
• Study First Submitted: 2011-06-07
• Study First Submitted QC: 2011-06-07
• Study First Posted: 2011-06-08
• Primary Completion: 2012-10
• Study Completion: 2013-03
• Status Verified: 2013-07
• Last Update Submitted: 2013-07-29
• Last Update Posted: 2013-07-30

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

1. Have histologically confirmed primary, recurrent or metastatic cerebral tumors and require chronic steroid dosing to manage symptoms of peritumoral brain edema (No histological confirmation is required of optic pathway tumors or brain stem gliomas)

2. Have been taking dexamethasone (or equivalent) at a dose of ≥1 mg/day for a minimum of 14 days and have had a stable dose for at least 7 days.

3. Have one or more steroid-associated side effects at Screening (See Appendix A1 for a list of qualifying events)

4. Capable of self-administration of subcutaneous injections twice daily or availability of assistance from caregiver.

5. Karnofsky/Lansky performance status ≥ 40

6. Life expectancy of at least 6 months

7. Female subjects must not be pregnant or breast-feeding and, if of childbearing potential, must use adequate methods of contraception during the study

8. Ability to provide written informed consent or, if unable to provide, have a legal guardian or representative provide written informed

Exclusion Criteria

1. Evidence of previous or current overt renal, hepatic or pulmonary diseases or active uncontrolled infection
2. Subject and/or parent/guardian is unwilling or unable to comply with this protocol
3. Subject has a known history of allergy or intolerance to hCRF or other ingredients in the Xerecept® solution (e.g., methionine, mannitol)
4. Subject has a known history of hepatitis C virus, hepatitis B surface, or human immunodeficiency virus (HIV).

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Xerecept	XERECEPT	All patients will receive hCRF (XERECEPT)

Primary Outcome Measures

Name	Time	Method
Maximum Tolerated Dose	2 months	To define the maximum tolerated dose (MTD) of Xerecept® in pediatric patients; the MTD will be defined as the dose at which 1 or fewer of six patients experiences a dose limiting adverse event of any kind.

Secondary Outcome Measures

Name	Time	Method
Dexamethasone Dosing	1 Year	To explore if Xerecept®, when administered to pediatric patients, is effective in allowing decreases in dexamethasone dosing
Incidence and severity of specified Steroid-Related Side Effects	1 year	To explore the clinical benefit associated with a reduction in steroid dosing over the course of the study by comparing the incidence and severity of pre-specified steroid-related side effects in all patients.
Number of patients with adverse events	1 Year	Adverse events will be recorded at each study visit and detailed by SOC to define the safety profile of Xerecept in pediatric patients
Change from baseline in clinical chemistry, hematology and urinalysis measures	1 Year	Standard clinical chemistry, hematology and urinalysis measures will be analyzed at baseline and monthly during the study; changes from baseline will be measured over the course of the study
PedsQL™ Quality of Life Inventory Scores	1 Year	Child self report and parent proxy reports conducted at each study visit to assess changes in health-related quality of life over the course of the study

Trial Locations

Locations (2)

Children's Memorial Hospital, Northwestern University Feinberg School of Medicine; 🇺🇸 Chicago, Illinois, United States

Dana-Farber Cancer Institute Pediatric Oncology; 🇺🇸 Boston, Massachusetts, United States